The multikinase inhibitor regorafenib significantly improved overall survival rates compared to placebo in patients with hepatocellular carcinoma, according to data from the phase 3 RESORCE trial presented at the ESMO 18th World Congress of Gastrointestinal Cancer.1

"Systemic treatment for hepatocellular carcinoma has long consisted of just one agent, sorafenib, which was shown to provide a significant improvement in life expectancy almost 10 years ago, but no other agent has surpassed its benefits," said Jordi Bruix, MD, head of the Barcelona Clinic Liver Cancer group at the Hospital Clínic in Spain and Scientific Director of the Network for Biomedical Research for Hepatic and Digestive Diseases (CIBEREHD), and principal investigator for the study.

This international, multicenter, phase 3 trial enrolled 573 patients with intermediate or advanced stage hepatocellular carcinoma, who had all been previously treated with sorafenib, and randomized them 2:1 to 160 mg oral regorafenib or placebo once daily for weeks 1 to 3 of each 4-week cycle, in addition to best supportive care.

After a median of 3.6 months of treatment, patients receiving regorafenib showed a 38% reduction in the risk of death and a 54% reduction in the risk of progression or death compared to placebo.

Patients had a mean progression-free survival of 3.1 months with regorafenib and 1.5 months with placebo, while median overall survival was 10.6 months for regorafenib and 7.8 months with placebo.

Overall, 65.2% of patients in the regorafenib group showed complete or partial response or stable disease, compared to 36.1% in the placebo group.

The safety and side effect profile of regorafenib was similar to sorafenib, with hypertension, hand-foot skin reaction, fatigue, and diarrhea all being significantly more common in patients taking the drug.

The benefits of the drug are evident regardless of the cause or stage of the tumor, Bruix explained, but analysis of biomarkers would reveal whether there might be certain subgroups of patients likely to derive even greater benefit from this treatment.

"This is a very difficult to treat cancer but now we have an effective second-line agent, which is good news for the patients and also for the field as interest in further developments will be stimulated," Bruix said.